Clinical trials: Muscle Atrophy
Limb-Girdle Muscular Dystrophy
Genetic Characterization of Individuals With Limb Girdle Muscular Dystrophy
The objective of this study is to identify and maintain a registry of well-characterized limb-girdle muscular dystrophy (LGMD) patients.
- Trial name
- NCT00457912
- Where
- United States
- Volunteers
- 277 people
- Sponsor
- Nationwide Children's Hospital
Duchenne Muscular Dystrophy
Outcome Measures in Duchenne Muscular Dystrophy: A Natural History Study
Novel emerging therapies for Duchenne Muscular Dystrophy (DMD) require a deeper understanding of DMD natural history. This study aim to assess the natural history of DMD through a composite assessment tool capable of capturing…
- Trial name
- NCT02780492
- Testing
- Set of assessment tools
- Where
- 5 sites in France, Netherlands and 1 more
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- University College, London
Muscular Dystrophy, Arthrogryposis
WREX Outcome Study
This study is to evaluate how the Wilmington Robotic Exoskeleton (WREX) is working for children who are using the WREX, or have used it in the past.
- Conditions
- Muscular Dystrophy, Arthrogryposis, Spinal Muscular Atrophy
- Trial name
- NCT02218593
- Testing
- WREX orthosis
- Volunteers
- 25 people
- Sponsor
- Nemours Children's Clinic
Duchenne Muscular Dystrophy, Cardiomyopathy
HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)
Male participants with cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) meeting all inclusion and no exclusion criteria will be randomized. All participants will be at least 12 years of age.
- Trial name
- HOPE (NCT02485938)
- Testing
- Allogeneic Cardiosphere-Derived Cells (CAP-1002)
- Where
- 3 sites in United States
- Volunteers
- 25 people
- Sponsor
- Capricor Inc.
Osteoporosis, Muscular Dystrophy
Assessment of Response to Treatment of Osteoporosis With Oral Bisphosphonates in Patients With Muscular Dystrophy
By supplying an adequate amount of calcium and vitamin D with the addition of weekly bisphosphonate, the investigators will be able to increase bone mass and decrease the incidence of fragility fractures in these children with…
- Conditions
- Osteoporosis, Muscular Dystrophy, Cystic Fibrosis
- Trial name
- NCT01882400
- Testing
- Bisphosphonate treatment
- Where
- Canada
- Volunteers
- 11 people
- Sponsor
- Gilles Boire
Rheumatoid Arthritis, Muscle Atrophy
Muscle Wasting and Rheumatoid Arthritis Flares
People living with Rheumatoid Arthritis (RA) often present with low muscle mass compared to their healthy counterparts. This affects their mobility, overall health and quality of life.
- Trial name
- NCT06400316
- Testing
- Amino Acid Supplement
- Volunteers
- 40 people
- Sponsor
- Leeds Beckett University
Duchenne Muscular Dystrophy
A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy
The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT07673809
- Testing
- GNR-097
- Where
- 6 sites in Belarus, Russia
- Volunteers
- 32 people
- Sponsor
- AO GENERIUM
Spinal Muscular Atrophy
Measuring Levels of SMN in Blood Samples of SMA Patients
Spinal muscular atrophy (SMA) is a disorder that affects the motor neurons. SMA is caused by a mutation in a part of the DNA called the survival motor neuron (SMN1) gene, which normally produces a protein called SMN.
- Trial name
- NCT00061607
- Where
- United States
- Volunteers
- 73 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Duchenne Muscular Dystrophy
Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
This is a Phase 2, open-label, single arm trial of pamrevlumab (FG-3019) to estimate pamrevlumab's safety and efficacy in non-ambulatory participants with DMD.
- Trial name
- MISSION (NCT02606136)
- Testing
- Pamrevlumab
- Where
- 10 sites in United States
- Volunteers
- 21 people
- Sponsor
- Kyntra Bio
Sarcopenia, Malnutrition
Nutritional Therapy in Patients at Risk for Malnutrition and Sarcopenia in Pulmonary Rehabilitation
Malnutrition and sarcopenia (muscle wasting) are common in health care settings and represent a health and economic burden due to associated increased mortality and prolonged hospital stays.
- Trial name
- NCT05096013
- Testing
- individual nutritional therapy
- Where
- Switzerland
- Volunteers
- 60 people
- Sponsor
- Thimo Marcin
Duchenne Muscular Dystrophy, Natural History
phenotypeS in Non Ambulant Duchenne Muscular Dystrophy
The aims of the study are to prospectively collect information on several aspects of function in non-ambulant DMD patients by using a structured battery of tests including motor, respiratory and cardiac function
- Conditions
- Duchenne Muscular Dystrophy, Natural History, Motor Function; Retardation
- Trial name
- GUP21003 (NCT06366815)
- Testing
- Different Phenotypes of non ambulant patients with Duchenne Muscular Dystrophy
- Where
- 2 sites in Italy
- Volunteers
- 250 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Spinal Muscular Atrophy
Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin America
The natural history of SMA patients has changed, due to the improvements in treatment and technological advances. The systematic collection of data from routine clinical practice in multiple Latin American countries, harmonized…
- Trial name
- RegistrAME (NCT05475691)
- Where
- 18 sites in Argentina, Brazil and 4 more
- Volunteers
- 361 people
- Sponsor
- Hospital Israelita Albert Einstein
Muscular Dystrophies, Muscle Inflammation
Safety and Efficacy of a Flavonoids- and omega3s-based Compound for the Treatment of Muscular Dystrophies
The study aimed to assess the safety and, partially, the efficacy of dietary supplementation of a flavonoids-, DHA- and EPA-based natural supplement in non-ambulant DMD boys and in a cohort of LGMD and FSHD patients to compare…
- Trial name
- PRO1 (NCT03317171)
- Testing
- Flavonoids, DHA, EPA
- Where
- Italy
- Volunteers
- 29 people
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Feasibility of Neonatal Screening for Spinal Amyotrophy
Feasibility of a Newborn Screening for Spinal Muscular Atrophy (SMA) in France: DEPISMA Prefigurator Project in Grand-Est and Nouvelle-Aquitaine
Spinal muscular atrophy (SMA) is a genetic disease of the nervous system that affects about 1 in 7,0001.2 births and results in very high mortality for patients with the disease.
- Trial name
- DEPISMA (NCT05645250)
- Where
- 39 sites in France
- Volunteers
- 220,000 people
- Sponsor
- University Hospital, Strasbourg, France
Osteoporosis, Duchenne Muscular Dystrophy
Effects of Low-level Mechanical Vibration on Bone Density in Ambulant Children Affected by Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a X-linked recessive disorder due to a mutation of the dystrophin gene (Xp21). Dystrophin is a sarcolemmal protein of skeletal and cardiac muscle, and its absence causes progressive muscle…
- Trial name
- NCT05281120
- Testing
- Low-level mechanical vibrations WITH vertical sinusoidal acceleration, Low-level mechanical vibrations WITHOUT vertical sinusoidal acceleration
- Volunteers
- 20 people
- Sponsor
- Istituto Auxologico Italiano
Muscular Dystrophies, Congenital Myopathy
Bullying in Youth With Muscular Dystrophy and Congenital Myopathies
Bullying is an epidemic in Canada, and rates may be underreported. Youth with a disability were more likely to be bullied that those without disabilities, specifically if the disability was visible.
- Trial name
- NCT04733976
- Where
- 2 sites in Canada
- Volunteers
- 29 people
- Sponsor
- Holland Bloorview Kids Rehabilitation Hospital
Sarcopenia, Aortic Valve Stenosis
Overal Survival After TAVI According to Sarcopenia
The goal of this observational study is to compare the overal survival after transcatheter aortic valve implantation (TAVI) according to sarcopenia status evaluated from preprocedural CT scans.
- Trial name
- TAVI_Sarx (NCT05672160)
- Testing
- CT evaluated sarcopenia
- Where
- Czechia
- Volunteers
- 930 people
- Sponsor
- Nemocnice AGEL Trinec-Podlesi a.s.
Limb-girdle Muscular Dystrophy
A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
This study will follow participants who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D (LGMD2D/R3), Limb-girdle muscular dystrophy…
- Trial name
- NCT04475926
- Where
- 26 sites in United States, Belgium and 8 more
- Volunteers
- 205 people
- Sponsor
- Sarepta Therapeutics, Inc.
Muscular Dystrophy, Facioscapulohumeral
Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
The primary goal of this study is to validate motor and functional outcomes and refine clinical trial strategies for pediatric-onset FSHD
- Trial name
- MOVE Peds (NCT06847282)
- Where
- 7 sites in United States, Australia
- Volunteers
- 80 people
- Sponsor
- University of Kansas Medical Center
Muscular Atrophy, Spinal
Progressive Strength Training in Spinal Muscular Atrophy
The proposed feasibility study is necessary to test if children and young adults will participate in and adhere to a 12-week, home-based, supervised progressive strength training exercise program and to obtain preliminary data…
- Trial name
- NCT01233817
- Testing
- Progressive strength training
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- University of Utah